Awarded To:  Queensland Facility for Advanced Bioinformatics (QFAB) 

Dates:  September 2014
Amount:  $14,300
 
The objective of this study was to identify gene expression differences in the blood of children with Angelman syndrome (AS) before, during and after minocycline treatment. To achieve this, monocytes were prepared from the blood of 8 AS children at 3 time points:
  1. baseline (0 weeks)
  2. after 8 weeks of treatment with minocycline.
  3. 8 weeks after the discontinuation of minocycline treatment (washout).

 

Background & Outcomes ~

In March 2013 24 children in the United States were recruited to "An open-label pilot trial of minocycline in children as a treatment for Angelman Syndrome". Prior to results being published, FAST Australia and the University of South Florida sought an Australian team to evaluate bloods from the clinical trial to determine if there were any changes in gene expression before, during and after the trial.

This data would allow us to determine a path forward in potential trials or investigations rather than investing in a duplicate study. Unfortunately the outcome of this study did not determine quantifiable changes in the samples citing low numbers and variability in the syndrome.


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